JP2018538287A5 - - Google Patents

Info

Publication number

JP2018538287A5

JP2018538287A5 JP2018529219A JP2018529219A JP2018538287A5 JP 2018538287 A5 JP2018538287 A5 JP 2018538287A5 JP 2018529219 A JP2018529219 A JP 2018529219A JP 2018529219 A JP2018529219 A JP 2018529219A JP 2018538287 A5 JP2018538287 A5 JP 2018538287A5

Authority

JP

Japan

Prior art keywords

mrna

protein

item

pharmaceutical composition

composition according

Prior art date

2016-12-13

Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)

Withdrawn

Links

• Espacenet
• Global Dossier
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• 108090000623 proteins and genes Proteins 0.000 claims description 112
• 108020004999 messenger RNA Proteins 0.000 claims description 110
• 102000004169 proteins and genes Human genes 0.000 claims description 101
• 230000000717 retained effect Effects 0.000 claims description 91
• 230000000692 anti-sense effect Effects 0.000 claims description 53
• 108020005067 RNA Splice Sites Proteins 0.000 claims description 48
• 239000008194 pharmaceutical composition Substances 0.000 claims description 38
• 108700028369 Alleles Proteins 0.000 claims description 31
• 230000000295 complement effect Effects 0.000 claims description 17
• 230000002950 deficient Effects 0.000 claims description 15
• 108020004707 nucleic acids Proteins 0.000 claims description 10
• 102000039446 nucleic acids Human genes 0.000 claims description 10
• 230000000694 effects Effects 0.000 claims description 8
• 150000007523 nucleic acids Chemical group 0.000 claims description 8
• 238000010255 intramuscular injection Methods 0.000 claims description 5
• 239000007927 intramuscular injection Substances 0.000 claims description 5
• 239000007928 intraperitoneal injection Substances 0.000 claims description 5
• 238000010253 intravenous injection Methods 0.000 claims description 5
• 238000004519 manufacturing process Methods 0.000 claims description 5
• 238000010254 subcutaneous injection Methods 0.000 claims description 5
• 239000007929 subcutaneous injection Substances 0.000 claims description 5
• 108091093037 Peptide nucleic acid Proteins 0.000 claims description 4
• 238000002347 injection Methods 0.000 claims description 4
• 239000007924 injection Substances 0.000 claims description 4
• 238000012986 modification Methods 0.000 claims description 4
• 230000004048 modification Effects 0.000 claims description 4
• 208000030761 polycystic kidney disease Diseases 0.000 claims description 4
• 238000012230 antisense oligonucleotides Methods 0.000 claims description 3
• 238000007913 intrathecal administration Methods 0.000 claims description 2
• 238000007914 intraventricular administration Methods 0.000 claims description 2
• 239000002243 precursor Substances 0.000 claims 4
• ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims 1
• 239000003814 drug Substances 0.000 claims 1
• 230000001105 regulatory effect Effects 0.000 claims 1
• RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
• 238000000034 method Methods 0.000 description 72
• 239000000203 mixture Substances 0.000 description 45
• 108091032973 (ribonucleotides)n+m Proteins 0.000 description 35
• 101001074439 Homo sapiens Polycystin-2 Proteins 0.000 description 20
• 101001026882 Homo sapiens Serine/threonine-protein kinase D2 Proteins 0.000 description 20
• 102100036142 Polycystin-2 Human genes 0.000 description 20
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• 108091027974 Mature messenger RNA Proteins 0.000 description 4
• FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 3
• 239000003085 diluting agent Substances 0.000 description 3
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• 102000004088 Proprotein Convertase 2 Human genes 0.000 description 1
• 108090000545 Proprotein Convertase 2 Proteins 0.000 description 1
• 210000003754 fetus Anatomy 0.000 description 1
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